Myotonic Dystrophy - Pipeline Insight, 2024

This “Myotonic Dystrophy- Pipeline Insight, 2024” report provides comprehensive insights about 24+ companies and 25+ pipeline drugs in Myotonic Dystrophy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Myotonic Dystrophy: Understanding

Myotonic Dystrophy: Overview

Myotonic Dystrophy (DM) is considered a subgroup of myopathy and the most common type of muscular dystrophy that begins in adulthood. There are two major forms recognized based on clinical and molecular presentation: Myotonic dystrophy type I (DM1), known as Steinert disease, and myotonic dystrophy type II (DM2), or proximal myotonic myopathy which is a milder variety of DMI. These are progressive, multisystem genetic disorders. Myotonia is characterized by impaired relaxation of muscles after voluntary contraction due to repetitive depolarization of the muscle membrane. Myotonia, due to myotonic dystrophy, improves with repeated exercise and is worsened by exposure to cold.

The pathophysiology of DMI is related to the number of CTG repeats, with more repeats correlating with more severe disease. Those with less than 35 repeats are considered normal, and those that manifest with clinical symptoms typically have greater than 50. Maternal transmissions will often result in greater CTG expansion. Additionally, the newly expanded alleles have a bias for further expansions that leads to the genetic phenomenon of anticipation when transmitted from one generation to the next.

Inactivation of the translation initiation factor eIF2A leads to general attenuation of translation. Sequestration of transcription factors and other nuclear factors also contributes to dysregulation of gene expression. Generation of small interfering RNA from sense (s) and antisense (as) DMPK transcripts might activate RNA interference pathways that lead to wider dysregulation of mRNA and protein amounts. sDMPK and asDMPK transcripts are subject to repeat-associated non-ATG translation in the polyglutamine (poly[Q]n) reading frame and possibly others, giving rise to toxic homopolymeric polypeptides that accumulate in the cytoplasm. Although some mechanisms have only been reported in myotonic dystrophy type 1, theoretically they could also operate in type 2 disease.

DM1 is caused by a change or alteration in the myotonic dystrophy protein kinase (DMPK) gene. DM2 is caused by a change or alteration in the nucleic acid-binding protein (CNBP) gene; this gene is also called the ZNF9 gene. Genes provide instructions for creating proteins that play a critical role in many functions of the body. When a mutation of a gene occurs, the protein product may be faulty, inefficient, or absent. Depending upon the functions of the particular protein, this can affect many organ systems of the body, including the brain. The change or alteration that affects these genes is called a repeat expansion. This means that a segment of DNA in the gene is repeated many times.

Myotonic Dystrophy are genetic disorders caused by localised mutations of DNA. Such DNA mutations result in a lack of dystrophin protein in DMD and an alteration of the protein production in DMs. No curative therapies are available to treat the pathogenic causes, and the identification of new therapeutic approaches to target genetic mutations is urgently needed. The most recent strategies to counteract MDs concern gene therapy and repurposing of drugs. Maximizing health and functional independence is the goal of supportive management. This is geared towards monitoring and treating all the medical issues associated with DM.

Physical and occupational therapy is recommended for strengthening weakened muscles, evaluation for orthotics, and durable medical equipment needs. Speech-language pathology (SLP) is required for dysphagia and swallowing studies or dysarthria as indicated. SLP is also utilized for intellectual disabilities and learning strategies.

Myotonic Dystrophy- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Myotonic Dystrophy pipeline landscape is provided which includes the disease overview and Myotonic Dystrophy treatment guidelines. The assessment part of the report embraces, in depth Myotonic Dystrophy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Myotonic Dystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Myotonic Dystrophy R&D. The therapies under development are focused on novel approaches to treat/improve Myotonic Dystrophy.

Myotonic Dystrophy Emerging Drugs Chapters

This segment of the Myotonic Dystrophy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Myotonic Dystrophy Emerging Drugs

Tideglusib: AMO PharmaAMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in additional CNS, neuromuscular and other orphan indications. AM0-02 is a clinical stage investigational medicine for the treatment of the severe form of congenital myotonic dystrophy known as DM1 or Steinert disease. In cellular and animal models of DM1 as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to increase.

AMO-02 is an inhibitor that has been shown to normalize levels of GSK3ß in transgenic models and in ex vivo tissue samples in patients with DM1 and to reduce levels of the mRNA that is pathogenic for DM1.Currently the drug is being investigated in Phase II/III stage of Clinical trial evaluation for the treatment of Congenital Myotonic Dystrophy.

Pitolisant: Harmony BiosciencesPitolisant (WAKIX) is a selective histamine 3 (H3) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H3 receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States. Currently the drug is being investigated in Phase II stage of Clinical trial evaluation for the treatment of Myotonic Dystrophy.

ENTR-701: Entrada TherapeuticsENTR-701, a proprietary Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer, is the second novel clinical candidate from Entrada’s growing pipeline of EEV therapeutics. ENTR-701 is designed to address the underlying cause of myotonic dystrophy type 1 through allele-specific targeting and blocking of the excess repeat-containing transcripts in dystrophia myotonica protein kinase mRNA. In doing so, ENTR-701 has the potential to restore the function of muscle blind-like proteins, correct the mis-splicing and aberrant expression of downstream transcripts and restore normal muscle function. Data from preclinical studies of ENTR-701 suggest correction of disease relevant biomarkers in various muscle groups. Currently, the product is in preclinical stage of development for the treatment of Myotonic dystrophy.

Myotonic Dystrophy: Therapeutic Assessment

This segment of the report provides insights about the different Myotonic Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Myotonic Dystrophy

• There are approx. 24+ key companies which are developing the therapies for Myotonic Dystrophy. The companies which have their Myotonic Dystrophy drug candidates in the most advanced stage, i.e. Phase II/III include, AMO Pharma.

Phases

This report covers around 25+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Myotonic Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Myotonic Dystrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Myotonic Dystrophy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Myotonic Dystrophy drugs.

Myotonic Dystrophy Report Insights

• Myotonic Dystrophy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Myotonic Dystrophy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Myotonic Dystrophy drugs?
• How many Myotonic Dystrophy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Myotonic Dystrophy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Myotonic Dystrophy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Myotonic Dystrophy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• AMO Pharma
• Harmony Biosciences
• Entrada Therapeutics
• Arrowhead Pharmaceuticals, Inc.
• Arthex Biotech
• NeuBase Therapeutics
• Enzerna

Key Products

• Tideglusib
• Pitolisant
• ENTR-701
• ARO-DM1
• ATX-01
• NT-0200
• NT 0231 F
• ENZ-001

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